Vertex announced results from two Phase 3 studies of lumacaftor in combination with ivacaftor in patients with cystic fibrosis (CF) who have two copies of F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

TRAFFIC and TRANSPORT were double-blind, placebo-controlled studies designed to evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in people with CF ages ≥12 who have two copies of the F508del mutation. Each study included two combination treatment groups and one placebo group. The combination treatment groups evaluated lumacaftor 600mg once daily or 400mg every 12 hours in combination with ivacaftor 250mg every 12 hours. Approximately 1,108 people were randomized to receive at least one dose of study drug in the two studies (549 in TRAFFIC; 559 in TRANSPORT). The primary endpoint of TRAFFIC and TRANSPORT was the mean absolute change from baseline in percent predicted FEV1 at the end of the 24-week treatment period as assessed by the average change in lung function at Week 16 and at Week 24. Based on the design of the study, which included multiple treatment arms within each study, statistical significance for each arm vs. placebo was based on a p-value of ≤0.0250.

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All four treatment arms within the studies met their primary endpoint. Additionally, statistically significant mean absolute and relative improvements in lung function were observed for all four treatment groups, both within group and vs. placebo, at all time points within the study (Weeks 2, 4, 8, 16 and 24). As patients in the study continued to be treated with their standard CF medicines, improvements observed for patients in the combination treatment arms were in addition to any benefits experienced with the use of other CF medicines. The mean baseline lung function of patients was approximately 61% predicted FEV1 for patients who received the combination regimen and for patients who received placebo. Mean absolute improvements in ppFEV1 of between 2.6 and 4.0 percentage points from baseline compared to placebo were observed across the studies (P≤0.0004), with mean relative improvements of 4.3–6.7% (P≤0.0007).

Based on these results, Vertex plans to submit regulatory applications for approval including a New Drug Application (NDA) in the fourth quarter of 2014 for people with CF ages ≥12 who have two copies of the F508del mutation.

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